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Agreements

Date: 2011-11-16

Type of information: R&D agreement

Compound: new cystic fibrosis drugs

Company: Genzyme (USA - MA), a Sanofi company (France) Cystic Fibrosis Foundation Therapeutics (USA)

Therapeutic area: Rare diseases - Genetic diseases

Type agreement: research - R&D

Action mechanism:

Disease: cystic fibrosis

Details:

  • Genzyme and Cystic Fibrosis Foundation Therapeutics, the nonprofit affiliate of the Cystic Fibrosis Foundation, have announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508. The program’s focus is to identify compounds known as “correctors,” which may aid in the ability of the malfunctioning CFTR protein found in CF patients to operate correctly. In the Delta F508 mutation, the CFTR protein does not move to its proper place at the cell surface, impeding the flow of fluids into the airways. Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.
  • In this collaboration, researchers will evaluate different compound libraries for correctors for Delta F508, and will take advantage of the vast compound libraries of both Genzyme and Sanofi. The research will take place throughout several Genzyme and Sanofi R&D facilities globally. Genzyme brings to the collaboration more than 20 years’ experience exploring treatments for people living with CF. The company’s efforts have ranged from improved molecular diagnostics to clinical trials with a gene therapy, and have included past collaboration with the CF Foundation in the area of drug discovery.

Financial terms:

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Is general: Yes